Clinical trials: Anemia, Hemolytic
Sickle Cell Disease
Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)
This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD).
- Trial name
- NCT03263559
- Testing
- Haploidentical Bone Marrow Transplantation, Hydroxyurea, Rabbit-ATG
- Where
- 32 sites in United States
- Volunteers
- 95 people
- Sponsor
- Medical College of Wisconsin
Sickle Cell Disease, Chronic Pain
Use of a Mobile-based App for SCD Patients
The study will seek to enroll 100 sickle cell or thalassemia patients who are age 12 or older who have access to a smartphone or tablet with Internet access daily.
- Trial name
- SMART (NCT01833702)
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Duke University
Sickle Cell Disease
Patient Characteristics and Treatment Patterns From Early Crizanlizumab Use in Real-world Setting: Preliminary Analysis From Select Sickle Cell Centers
This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with at least five patients who initiated crizanlizumab.
- Trial name
- NCT05833022
- Where
- 11 sites in United States
- Volunteers
- 297 people
- Sponsor
- Novartis Pharmaceuticals
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders
This clinical trial is studying how well giving fludarabine phosphate and melphalan together with total-body irradiation followed by donor stem cell transplant works in treating patients with hematologic cancer or bone marrow…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00856388
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Roswell Park Cancer Institute
Acute Chest Syndrome, Sickle Cell Disease
Ultrasound Acute Chest Syndrome Sickle Cell Disease
Feasibility and reliability of ultrasound in the inpatient hematology setting.
- Trial name
- POCUS (NCT06880679)
- Testing
- Point of Care Ultrasound
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Indiana University
Immune Thrombocytopenia, Autoimmune Hemolytic Anemia
Severe Immune Cytopenia Registry Www.Sic-reg.Org
Prospective registry study for children and young adults with severe immune cytopenias (persisting/chronic immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome) to improve the management, facilitate the…
- Conditions
- Immune Thrombocytopenia, Autoimmune Hemolytic Anemia, Evans Syndrome, Primary Immunodeficiency
- Trial name
- sic-reg (NCT03576742)
- Testing
- potential biomarkers
- Where
- Austria
- Volunteers
- 80 people
- Sponsor
- Medical University of Graz
Beta-Thalassemia Major, Graft-versus-host Disease (GVHD)
Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major
The purpose of this study is to evaluate the basiliximab for prevention of graft-versus-host disease in unrelated allo-genetic hematopoietic stem cell transplantation for thalassemia major.
- Trial name
- NCT02342145
- Testing
- Basiliximab,, Tacrolimus, Methotrexate
- Where
- 3 sites in China
- Volunteers
- 205 people
- Sponsor
- Zhongming Zhang
Sickle Cell Disease, Vaso-occlusive Crisis
Steroid Treatment for Sickle Cell Pain Crisis
The painful episode is the most common problem experienced by children with sickle cell disease. Although various treatments are available during painful episodes, the medication most commonly given for pain is a pain medication…
- Trial name
- NCT00263562
- Testing
- Steroid arm
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Baylor College of Medicine
Thalassemia, Brain Function (Measured by ERP Level)
A Case Control Study to Evaluate the Cognitive and Brain Function of β-thalassemia Patients.
β-thalassemia syndromes are a group of hereditary disorders characterized by a genetic deficiency in the synthesis of beta-globin chains.
- Trial name
- NCT01935661
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- HaEmek Medical Center, Israel
Sickle Cell Anemia
Sickle Cell Disease: A Retrospective Chart Review
This study is a retrospective chart review of sickle cell patients and will include patients whom have received blood transfusions and those whom have not.
- Trial name
- NCT01441375
- Where
- 2 sites in United States
- Volunteers
- 261 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease
The purpose of this study it to evaluate a reduced toxicity conditioning regimen for haploidentical donor HCT followed by a GVHD prophylaxis regimen comprising of post-transplant cyclophosphamide, sirolimus and abatacept with the…
- Trial name
- NCT07616154
- Testing
- Cyclophosphamide 50mg, Abatacept, Alemtuzumab
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- St. Jude Children's Research Hospital
Anemia, Sickle Cell, Blood Disease
Cooperative Study of The Clinical Course of Sickle Cell Disease
To determine the natural history of sickle cell disease from birth to death in order to identify those factors contributing to the morbidity and mortality of the disease.
- Trial name
- NCT00005277
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Thrombotic Thrombocytopenic Purpura, Atypical Hemolytic Uremic Syndrome
TTP and aHUS in Complicated Pregnancies
A single site observational study aiming to: (i) Identify cases of previously undiagnosed thrombotic thrombocytopenic purpura (TTP) and atypical haemolytic syndrome (aHUS) in a cohort of women with complicated pregnancies (ii)…
- Conditions
- Thrombotic Thrombocytopenic Purpura, Atypical Hemolytic Uremic Syndrome, Pre-Eclampsia, HELLP Syndrome
- Trial name
- NCT03605511
- Testing
- ADAMTS 13, complement and angiogenic biomarkers
- Where
- United Kingdom
- Volunteers
- 150 people
- Sponsor
- University College, London
Sickle Cell Disease, Asthma
Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial
Acute and chronic pulmonary complications with concomitant inflammatory response are a leading cause of morbidity and mortality in children with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Asthma, Acute Chest Syndrome
- Trial name
- NCT02187445
- Testing
- Budesonide inhalation suspension
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Vanderbilt University
Sickle Cell Disease, Vaso-Occlusive Crisis
Adjuvant Low-dose Ketamine in Pediatric Sickle Cell Vaso-occlusive Crisis
Acute vaso-occlusive episodes (VOEs) in sickle cell disease (SCD) are primarily managed with opioids. Tolerance and hyperalgesia to opioids develops due to N-methyl-D-aspartate (NMDA)-receptor mediated activation of the…
- Trial name
- AKTSS (NCT03296345)
- Testing
- Ketamine
- Where
- United States
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Disease
Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare…
- Trial name
- NCT05810181
- Testing
- Interview
- Where
- United States
- Volunteers
- 145 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
A study designed to evaluate the non-inferiority of crovalimab compared with eculizumab in participants with PNH who have not been previously treated with complement inhibitor therapy.
- Trial name
- COMMODORE 2 (NCT04434092)
- Testing
- Crovalimab, Eculizumab
- Where
- 63 sites in Argentina, Brazil and 21 more
- Volunteers
- 210 people
- Sponsor
- Hoffmann-La Roche
Sickle Cell Disease (SCD), Invasive Bacterial Infections
Epidemiology of Invasive Bacterial Infections in Children With Sickle Cell Disease in France Between 2020 and 2025
Children with sickle cell disease are at high risk of invasive bacterial infections, which may lead to serious complications. Preventive measures, including antibiotic prophylaxis and vaccination, have changed the epidemiology of…
- Trial name
- DREPIBAC (NCT07639008)
- Where
- 31 sites in France
- Volunteers
- 350 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Assessing Function in Pediatric Patients With Sickle Cell Disease
The purpose of this study is to evaluate the FIM™ as a measure of daily function in children with sickle cell disease hospitalized with vasoocclusive pain.
- Trial name
- NCT00590148
- Where
- 2 sites in United States
- Volunteers
- 25 people
- Sponsor
- Connecticut Children's Medical Center
Sickle Cell Anemia
Neutrophil Extracellular Traps and Sickle Cell Disease
NETs formation in vaso-occlusive events in sickle cell disease and the role of hydroxyurea treatment.The study hypothesis is that NETs formation by neutrophils has a role in the induction of vaso occlusive disease in blood…
- Trial name
- NCT02004808
- Volunteers
- 15 people
- Sponsor
- Tel-Aviv Sourasky Medical Center
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